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TGDOC Newsletter Winter 2021 ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏ ͏
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TGDOC Annual Curators Meeting 2020
Thank you to all TGDOC Members who attended the TGDOC Annual Curators Meeting between 30th November and 2nd December 2020. Held online due to the Coronavirus pandemic, this year’s meeting was bigger than ever, with 132 registered attendees participating in 24 individual sessions, as well as an online quiz and networking time.
During the meeting, we welcomed Michela Guglieri as the new TGDOC Incoming Chair gave a fond farewell to Nathalie Goemans who has now reached the end of her six years on the TGDOC Chair troika. The entire TGDOC community would like to thank Nathalie for her dedication and hard work over the years and wish her a happy and relaxing retirement.
Special thanks also to our guest speakers Durhane Wong-Rieger (Canadian Organization for Rare Disorders) and Elizabeth Vroom (World Duchenne Organisation) who delivered the ‘Hearing the Patient Voice in Rare Disease Organizations’ session, and also to Jim Hilbert (Rochester National Registry for DM & FSHD) and Marjan Cosyns (Belgian Neuromuscular Diseases Registry) who provided insights into their own registries in the Registry Spotlight sessions.
In addition to dedicated disease subgroup breakout sessions, the meeting also included sessions on the new TGDOC Membership Process and TGDOC Publications Committee, an overview of completed Registry Enquiries and responses to the Registry Review Surveys. Updates were also provided on the Core Dataset projects for DMD, SMA and LGMD, and the Universal Registries Platform (URP) and EMA qualification process.
All sessions were recorded and are available to view on the Whova meeting app (please note the session recordings are not available publicly and can only be accessed by registered TGDOC Members with a Confidential Disclosure Agreement in place). A link to the full meeting report will be circulated to TGDOC Registries in the coming weeks and an abridged version will be made available to all on the TREAT-NMD website.
Thank you to everyone who has already submitted feedback following the meeting, the feedback we have received so far has been overwhelmingly positive. If you attended the TGDOC Curators’ meeting and have not yet submitted your feedback form it is not too late to contribute, please complete the survey here.
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Has your registry completed the TGDOC Membership Pack?
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Following a summer of reflection and consultation with the TGDOC community and TREAT-NMD Executive Committee, the TGDOC Membership Process was launched in October 2020. All new and existing TGDOC registries should have now begun this process. If your registry has not yet started this process we strongly encourage you to download and complete the TGDOC Membership Pack as soon as possible. This includes everything you will need to confirm your membership, including checklists for Core and Affiliated Membership status and a new CDA agreement with TREAT-NMD.
Once your completed Membership Pack has been processed by the TREAT-NMD Secretariat, you will receive your Membership Certificate and a personalised Welcome Pack with information tailored to your registry. We are very grateful to all the registries who have already completed or are in the final stages of this process.
If you have any questions about the new TGDOC Membership Process that are not answered in the Membership Pack, please don’t hesitate to get in touch with Helen or Emma.
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Volunteers needed for the TGDOC Publications Committee
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We are looking for volunteers to join the TGDOC Publications Committee.
The TGDOC Publications Committee performs several functions to support TGDOC Member Registries and ensure the high-quality output of publications using collective TGDOC registry data. Further information can be found on the Publications Committee webpage.
If you would like to put yourself forward for consideration, please contact Dr Rasha El Sherif.
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Key changes to the TREAT-NMD SMA Core Dataset structure
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The TREAT-NMD SMA Core Dataset project held its annual curators’ workshop at the end of October 2020 where the focus was on presenting and discussing the version 2 dataset, released earlier that month. Marcel Heidemann provided a comprehensive demonstration and explanation of the key structural changes from v1 to v2, including the following topics: - Naming and wording conventions
- Data structures; items and records
- Unique data item identifiers
- Data descriptions and definitions
- Datestamp requirements and collecting/storing longitudinal data
- Interactive sample questions and example data representations
A full recording is available via this video.
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Empowering patient involvement in registry and PROM development
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- Do you have any experience of successfully integrating the patient voice into the design and planning of data collection?
- Have you learnt lessons that others could benefit from?
- Can you speak from personal experience about how a patients' direct experience of a disease can complement the clinical and scientific knowledge of other experts?
As part of the SMA Core Dataset work, SMA Europe and TREAT-NMD have identified a need to empower and inspire the patient community to be more involved in all stages of data collection related to their neuromuscular disease. We are planning a series of workshops and we are looking for speakers for the first one, tentatively scheduled for mid-April. The intended outcome of this workshop would be a motivated group of SMA, who understand their role and the value of their perspective, and are confident and ready to contribute to registry and PROMS development. If successful, we would hope to open this up to other NM disease areas in future. If you think you could help by speaking at this workshop, please let us know by completing this short form before Wednesday 10th February 2021.
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TREAT-NMD Universal Registry Platform
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We are now halfway through the Universal Registry Platform (URP) Project and we are making good progress.
The Clinician Portal build was completed in mid-December 2020 and was ready for Beta testing as part of the project programme. The Beta testing was undertaken by a selection of DMD and SMA Registries, who were provided access to the system and began testing the week before Christmas. The Registries were asked to provide all testing feedback by the close of business on the 22nd January 2021 and that feedback is currently being collated, it will then be analysed internally by the URP project team before any change requests or additions to the system are proposed. Meetings will be held in early February to discuss the testing feedback in more detail to agree any changes and these meetings will be with the IT contractors, Dataset Project managers and TGDOC Chairs.
The Patient Portal build began at the beginning of January and development is currently underway with updates being provided at the fortnightly ‘sprint’ meetings. As part of the Patient Portal build, we are also looking at Dual Reported Registries and gathering information to ensure that this element of the URP meets the needs of its users. We are anticipating being able to issue the Patient Portal for Beta testing by a selection of DMD and SMA Registries at the end of March.
In addition to the Clinician and Patient Portal for SMA and DMD disease modules, we have now received the confirmation of funding for adding a further disease module to the URP for Limb Girdle Muscular Dystrophy (LGMD). This is exciting news and as the build for the LGMD module will begin almost 5 months ahead of the intended project start date, it is a great show of confidence in the URP and will be of great value to the LGMD registries, many of whom do not have robust systems of their own.
On a final note, the URP team is growing and we will be welcoming two new team members at the end of January to assist with the planning, roll out and implementation of the URP across the TGDOC registry network.
For further information on the URP or to participate as a ‘test registry’ then please contact Caroline Ogden, the URP Project Manager or visit our website to learn more about the URP
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Parent Project’s International Conference is coming (online)
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The 18th edition of Parent Project’s International Conference on Duchenne and Becker Muscular Dystrophy will take place online from the 17th to the 21st of February, 2021.
Last year's edition had to be cancelled due to the Covid-19 outbreak; this year the event will be back, although based on a web platform, to provide up-to-date scientific information and much more for the entire Duchenne and Becker community.
The 2021 conference claim will be "Alone we imagine the future, together we grow" with the subtitle "Many paths of research towards one direction", to underline the fundamental dynamics of convergence and interrelation between the many actors who make up the Duchenne community.
The most recent updates on research and clinical studies will be dealt with in scientific sessions that will also embrace the various aspects of the clinical management of the disease. These will alternate with parallel sessions on issues related to daily life (from patients’ rights to affectivity and sexuality, to the role of siblings and more) and sessions dedicated to psychosocial aspects, with direct testimonies from the community of patients and families. In addition, some moments dedicated to socializing will be included in the programme. A translation service (in English) will be available for all the sessions.
In a few days it will be possible to download the draft agenda and to register for the Conference following the instructions on Parent Project's website.
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